Gene therapy trial Via Copernicus Therapeutics; copied by n

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Espicom Business Intelligence

April 30, 2003

HEADLINE: CF gene therapy trial results encouraging

Scientists and physicians in Cleveland have announced encouraging

results

from the first-of-its-kind gene therapy trial involving cystic fibrosis

(CF)

patients and a new compacted DNA technology. The Phase I trial involving

12

patients was launched a year ago by University Hospitals of Cleveland

(UHC),

Case Western Reserve University (CWRU) School of Medicine, Children's

Hospital

of Denver and Cystic Fibrosis Foundation Therapeutics.

Copernicus Therapeutics produced the non-viral gene transfer system

used in

the clinical trial. Working together with UHC and CWRU researchers,

Copernicus

formulated a way to 'compact' or tightly bind strands of DNA so that it

is tiny

enough to pass through a cell membrane and into the nucleus. The ultimate

goal

is for the DNA to produce a protein needed by people with CF to correct

the

basic defect in CF cells.

The primary goal of this Phase I study was to determine if this gene

therapy

method is safe and tolerable as administered in the trial. All

participants

completed the trial without significant side effects and the treatment

itself

was well tolerated. The secondary goals of the trial were to evaluate if

the CF

gene was successfully transferred to airway cells and if it functioned

normally,

results which would suggest that this therapy may be of benefit to people

with

CF. According to the researcher, the data were very encouraging with

indications

that this gene transfer may have occurred.

The company is currently working on an aerosol version that would

enable

people with CF to have the healthy gene delivered directly into their

lungs.

During the recently completed study, patients received the compacted DNA

in a

saline solution dripped into the nasal passages. Future clinical trials

will

study the safety and efficacy of the aerosol approach.

The research teams delivered the healthy gene into 12 adult CF

patients in a

saline solution dripped slowly into their nasal passages. Investigators

monitored salt transport in the nose (nasal potential difference) as a

barometer

of the procedure's success. CF patients have a markedly abnormal nasal

potential

difference. Through biopsies of nasal tissue, researchers determined

whether the

healthy gene was taken up by the cells and produced enough protein to

affect the

transport of salt and water in and out of the cells. They found that

two-thirds

of patients treated had a meaningful increase in the transport of

chloride ion

in the nose.

The researchers and physicians involved in this study will present

their

findings at the American Society of Gene Therapy meeting, to be held from

4th to

8th June, in Washington, DC, and at the Cystic Fibrosis Foundation

burg

meeting, to be held form 31st May to 4th June.